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Optimal two-stage designs for phase II clinical trials.

Author information

Affiliations

  • 1. Biometric Research Branch, National Cancer Institute, Bethesda, Maryland 20892.
      Authors
    • Simon R 1
    (1 author)

Controlled Clinical Trials, 01 Mar 1989, 10(1):1-10
https://doi.org/10.1016/0197-2456(89)90015-9 PMID: 2702835 

Abstract 

The primary objective of a phase II clinical trial of a new drug or regimen is to determine whether it has sufficient biological activity against the disease under study to warrant more extensive development. Such trials are often conducted in a multi-institution setting where designs of more than two stages are difficult to manage. This paper presents two-stage designs that are optimal in the sense that the expected sample size is minimized if the regimen has low activity subject to constraints upon the size of the type 1 and type 2 errors. Two-stage designs which minimize the maximum sample size are also determined. Optimum and "minimax" designs for a range of design parameters are tabulated. These designs can also be used for pilot studies of new regimens where toxicity is the endpoint of interest.

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Read article at publisher's site: https://doi.org/10.1016/0197-2456(89)90015-9

Read article for free, from open access legal sources, via Unpaywall: http://linus.nci.nih.gov/techreport/Optimal2-StageDesigns.pdfUnpaywall

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